Release Date: August 01, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Alnylam Pharmaceuticals Inc (ALNY, Financial) achieved 34% year-over-year growth in global net product revenues, generating $410 million in net product revenues.
- The company raised its revenue guidance for the year by 11% at the midpoint.
- Positive top-line results from the Helios Phase III study of vutrisiran in ATTR cardiomyopathy, showing a 35% to 36% mortality benefit compared to placebo.
- Strong growth in the TTR franchise, with $307 million in global net product revenues, representing a 37% increase compared with the second quarter of 2023.
- Alnylam Pharmaceuticals Inc (ALNY) ended the second quarter with cash, cash equivalents, and marketable securities of $2.6 billion, up from $2.4 billion as of December 31, 2023.
Negative Points
- Non-GAAP R&D expenses increased by 14% in the second quarter compared to the same period in the prior year.
- Non-GAAP SG&A expenses increased by 21% in the second quarter compared to the same period in the prior year.
- Gross margin on product sales is expected to be lower for the balance of the year due to higher royalties paid on vutrisiran.
- The opt-out of mybelt soran by Regeneron will result in increased R&D investment as the program is now wholly owned by Alnylam Pharmaceuticals Inc (ALNY).
- The company is increasing its planned ATTR cardiomyopathy launch prep efforts, which will drive up expenses.
Q & A Highlights
Q: Can you discuss the reimbursement landscape in ATTR cardiomyopathy and how you're thinking about the potential difference in pricing between polyneuropathy and cardiomyopathy?
A: Tolga Tanguler, Chief Commercial Officer, emphasized the company's strong commercial performance and the seamless access patients have to therapy. He highlighted the advantage of being physician-administered under Medicare Part B, which allows for better reimbursement dynamics compared to other products.
Q: What feedback have you received from physicians about the expectations for vutrisiran in first-line treatment based on the Helios B results?
A: Pushkal Garg, Chief Medical Officer, noted that physicians are enthusiastic about the potent treatment option vutrisiran offers. The Helios B study showed significant efficacy, making vutrisiran well-positioned to be a first-line agent in treating ATTR cardiomyopathy.
Q: Will you report KM curves and time-to-event kinetics for the different subgroups in the Helios B study at the ESC conference?
A: Pushkal Garg confirmed that the presentation would include detailed results, including baseline characteristics, primary and secondary endpoints, and consistency of effects across various subgroups. The data will provide a comprehensive view of the study's outcomes.
Q: Can you provide more details on the potential Phase II design for ALN-APP in Alzheimer's disease and the status of the HBNE obesity program?
A: Pushkal Garg mentioned that while specific details on the Phase II design for ALN-APP in Alzheimer's disease are still being finalized, the program remains on track. He also noted that the company is aggressively pushing forward with multiple programs, including the HBNE obesity program.
Q: What are your thoughts on the real-world data showing disease progression stabilization in patients switching from tafamidis to patisiran?
A: Pushkal Garg highlighted that the real-world data is encouraging and underscores the unmet need for additional therapies in treating ATTR polyneuropathy and cardiomyopathy. The data supports the potential for vutrisiran to address this need effectively.
Q: What level of detail should we expect in the interim results for Part B of the Phase I ALN-APP study later this year?
A: Pushkal Garg indicated that the interim results would focus on safety and pharmacodynamics with multiple dosing. The company is excited about the potential of ALN-APP and will share more data as it becomes available.
Q: Have the nature of discussions with physicians changed since the launch of eplontersen, and how is the market evolving with the new competitive therapy?
A: Tolga Tanguler noted that the market is evolving positively, with increased promotional share of voice and a clear differentiation of product profiles. The company continues to see rapid growth and expects to maintain momentum.
Q: Can you discuss the patient population and geographic locations for vutrisiran's reimbursement patterns?
A: Tolga Tanguler explained that the company adheres to a patient access philosophy that minimizes co-pay burdens. Nearly 70% of patients have zero co-pay, and up to 80% pay less than $2,000. This approach has been effective in both the U.S. and global markets.
Q: How are you positioning vutrisiran for TTR-CM, and what are your commercial strategies for scaling up the sales force?
A: Tolga Tanguler emphasized the company's strong foundation in TTR centers and integrated customer-facing teams. The focus is on scaling up capabilities and ensuring seamless patient access and reimbursement. The company is also preparing for label expansion and effective communication of the product's profile.
Q: What are the key factors contributing to 80% of patients with TTR-CM going untreated, and how do you plan to address this?
A: Tolga Tanguler and Pushkal Garg highlighted the need for increased disease awareness and education. The company is engaging with centers to improve early diagnosis and treatment, leveraging non-invasive diagnostic methods and red flag symptoms to identify patients earlier.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
